OVERVIEW

There is a significant unmet medical need for an oral drug that is safe and effective in the treatment a spectrum of autoimmune diseases that includes psoriasis, ankylosing spondylitis and inflammatory bowel disease.

Anti-TNFα and other biologic approaches to the treatment of autoimmune disease are limited by significant side effects and cost.

The devastating pathology associated with autoimmune diseases is caused by inappropriate activation of the immune response. The two key T cell types involved in disease are Th17 cells and innate lymphoid cells, both of which produce large amounts of inflammatory cytokines, most notably interleukin 17.

The “holy grail” in this therapeutic area is to identify a therapeutic agent that will control the activity of those T lymphocytes that are responsible for the disease (Th17 cells and innate lymphoid cells), while retaining the desirable responses driven by other T cells (Th1), which are needed for clearance of infections, particularly those of viral origin.

RORγt is an orphan nuclear hormone receptor that plays a critical role in the control of genes responsible for the differentiation and activation of Th17 and innate lymphoid cells.